A small n sequential multiple assignment randomized trial design for use in rare disease research.

ABSTRACT:

Background

Clinical trials in rare diseases are difficult to conduct due to the limited number of patients available with each disorder. We developed a Phase 2 trial which is a small n sequential multiple assignment randomized trial (snSMART) design to test several treatments for a rare disease for which no standard therapy exists.

Purpose

This paper illustrates the design, sample size estimation and operating characteristics of an snSMART.

Methods

We investigate the performance of a class of weighted Z statistics via computer simulations.

Results

We demonstrate the increase in power over traditional single stage designs, and indicate how the power changes as a function of the weight given to each stage.

Conclusion

The snSMART design is promising in a ra

SUBMITTER: Tamura RN 

PROVIDER: S-EPMC4695231 | biostudies-literature | 2016 Jan

REPOSITORIES: biostudies-literature